RecruitingPHASE3INTERVENTIONAL

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Age With Classical Homocystinuria (HCU) Due to Cystathionine Beta Synthase Deficiency Receiving Standard of Care Treatment

This study is investigating a new medication called pegtibatinase for individuals aged 12 to 65 who have a rare genetic condition called Homocystinuria (HCU). HCU means the body struggles to break down certain proteins. The study will compare pegtibatinase to a placebo (a dummy drug) to see how well it works and if it's safe. Participants will continue their usual HCU treatments throughout the study. The main goal is to find out if pegtibatinase can help manage HCU more effectively. This is a "Phase 3" study, meaning it's one of the final steps before a medicine might be approved for wider use.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Travere Therapeutics, Inc.

Enrolment target

Start

28 Dec 2023

Estimated completion

01 Sep 2027

Homocystinuria

What is this study about?

This research is looking into a new treatment called pegtibatinase for a condition called Homocystinuria (HCU). HCU is a rare inherited condition where your body has trouble processing a building block of protein called methionine. This can lead to a harmful substance called total homocysteine (tHcy) building up in your blood, which can cause serious health problems if not managed.

The main aim of this study is to see if pegtibatinase is effective and safe for people with HCU. It will compare the new drug with a placebo, which looks just like the study drug but doesn't contain any active medicine. This is a common way to test new treatments, as it helps researchers understand if any changes are truly due to the new drug. All participants in the study will continue to receive their standard HCU care, such as special diets and other medications, alongside the study treatment.

This study is a 'Phase 3' trial, which means it's a large, important step in testing a new medicine. If the results are positive, it could lead to pegtibatinase becoming a new treatment option for people with HCU. The study is international and will involve around 70 people to get a good understanding of how the drug works.

Key takeaways

This study is testing a new drug, pegtibatinase, for Homocystinuria (HCU).
It aims to find out if the drug is both safe and works better than a dummy treatment (placebo).
Participants will continue their usual HCU diet and treatments.
The study lasts up to 38 weeks and involves regular check-ups.
You will not know if you're getting the active drug or placebo during the main treatment period.

Who may be eligible?

To join this study, you need to be between 12 and 65 years old and have a confirmed diagnosis of Homocystinuria (HCU). Your blood test for total homocysteine (tHcy) needs to show levels of 50 µM (micromoles per litre) or higher. It's important that you're willing to stick to a stable diet and continue your current HCU treatments (like betaine or special medical foods) throughout the study, unless your doctor says otherwise for your health.

There are also some reasons why you might not be able to join. For example, if you have certain other medical conditions (like Marfan syndrome or recent serious blood clots), or if you've had a bad reaction to similar drugs in the past. If you weigh more than 160 kg, or if you've previously taken pegtibatinase or similar study drugs, you wouldn't be able to participate. If you could become pregnant, you'll need a negative pregnancy test and to use effective contraception.

Quick self-check

Are you between 12 and 65 years old?
Do you have a confirmed diagnosis of Homocystinuria (HCU)?
Is your homocysteine level typically 50 µM or higher?
Are you willing to stick to a stable diet and your current HCU treatments during the study?
Have you NOT had a serious blood clot in the last 6 months?
Have you NOT taken pegtibatinase or similar study drugs before?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to take part, the study will last up to 38 weeks (about 9 months). It starts with a screening period of up to 10 weeks to check if you're suitable. This includes an initial check-up and a period of up to 6 weeks where your diet will be carefully monitored to make sure it's consistent. During this time, your regular HCU diet and treatments will be recorded.

After screening, you'll enter the 'blinded treatment period' for 24 weeks. This means you won't know if you're receiving pegtibatinase or the placebo, and neither will your study doctor. This helps to ensure the results are accurate. You'll have regular study visits, some at home and some possibly online, where your diet and HCU treatments will continue to be monitored. After your last dose of the study medicine, there will be a 4-week safety check-up, unless you choose to join a long-term follow-up study.

Potential risks and benefits

Taking part in any study has potential benefits and risks. A potential benefit could be that pegtibatinase helps to manage your HCU more effectively than your current treatment, which could improve your health. However, there's also a chance you might receive the placebo and not experience any direct benefits from the study drug. Risks could include side effects from the medication or the inconvenience of study visits and dietary monitoring. It's important to remember that you can stop participating in the study at any time, for any reason, without it affecting your usual medical care.

Locations (52)

Phoenix Children's Hospital
Phoenix, United States· Not yet recruiting
Yale University School of Medicine
New Haven, United States· Recruiting
Emory University
Atlanta, United States· Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, United States· Not yet recruiting
Maine Health - Maine Medical Center
Portland, United States· Recruiting
Uncommon Cures
Chevy Chase, United States· Recruiting
The Mount Sinai Hospital
New York, United States· Recruiting
Science 37 - Virtual Site
Morrisville, United States· Recruiting
Oregon Health and Science University
Portland, United States· Not yet recruiting
Children's Hospital of Philadelphia
Philadelphia, United States· Recruiting
University of Pittsburgh Medical Center - Children's Hospital of Pittsburgh
Pittsburgh, United States· Recruiting
University of Texas Southwestern Medical Center
Dallas, United States· Recruiting

+40 more sites — see the official record for the full list.

Common questions

What is Homocystinuria (HCU)?

HCU is a rare genetic condition where your body has trouble processing certain proteins, leading to a build-up of a harmful substance called homocysteine in your blood.

What is a placebo?

A placebo is a 'dummy' drug that looks exactly like the real medicine but doesn't contain any active ingredients. It's used to compare the new drug's effects accurately.

Will I know if I'm getting the real drug or the placebo?

No, during the 24-week treatment period, neither you nor your study doctor will know if you're receiving pegtibatinase or the placebo. This is called 'blinded' and helps ensure fair results.

How long will I be in the study?

The total study duration is up to 38 weeks, which includes screening, diet standardisation, and 24 weeks of active treatment.

Can I continue my usual HCU treatments during the study?

Yes, you will continue your standard HCU care, including your special diet and any other HCU medications, throughout the study.

How to find out more

Travere Call Center

medinfo@travere.com 1-877-659-5518 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.